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Key information relevant to the recruitment process for the
overall study, such as dates of the recruitment period and locations

A total of 252 participants were randomized into the double-blind phase of the study across 15 countries. They were randomized 2:1 in a blinded manner to receive pomalidomide 0.5 mg/day or matching placebo capsule/day.

Pre-Assignment Details

Significant events and approaches for the overall study
following participant enrollment, but prior to group assignment

Participants had myeloproliferative neoplasm (MPN)-associated myelofibrosis defined as primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis and were red blood cell (RBC) transfusion-dependent having an average RBC-transfusion-frequency of ≥2 units of RBC every 28 days over at least 84 days.

Explanation of how the number of participants for analysis was determined.
Includes whether analysis was per protocol, intention to treat, or another method.
Also provides relevant details such as imputation technique, as appropriate.

Intent to Treat (ITT) population included all randomized participants

Reporting Groups

Description

Pomalidomide 0.5 mg (Oral Capsule)

Pomalidomide 0.5 mg by mouth daily Days 1 through Day 168

Placebo (Oral Capsule)

Matching oral placebo Days 1 through Day 168

Total

Total of all reporting groups

Baseline Measures

Pomalidomide 0.5 mg (Oral Capsule)

Placebo (Oral Capsule)

Total

Overall Participants Analyzed [Units: Participants]

168

84

252

Age [Units: Years]Mean (Standard Deviation)

68.0 (8.89)

68.6 (8.16)

68.2 (8.64)

Sex: Female, Male [Units: Participants]Count of Participants

Female

41 24.4%

28 33.3%

69 27.4%

Male

127 75.6%

56 66.7%

183 72.6%

ECOG Performance Status [1] [Units: Participants]

0 = (Fully Active)

53

22

75

1 = (Restrictive but ambulatory)

85

47

132

2 = (Ambulatory but unable to work)

30

15

45

3 = (Limited self care)

0

0

0

4 = (Completely Disabled)

0

0

0

[1]

ECOG-Eastern Cooperative Oncology Group (ECOG) Performance Status is used by doctors and researchers to assess how a participant's disease is progressing, assess how the disease affects the daily living activities of the participant and determine appropriate treatment and prognosis.

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release
and can embargo communications regarding trial results for a period that is less than or equal to 60 days.
The sponsor cannot require changes to the communication and cannot extend the embargo.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release
and can embargo communications regarding trial results for a period that is more than 60 days but less than
or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.

Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.

Restriction Description:
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1 year since study completion. Then Investigator can publish if the manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides a publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission or defer publication to permit patent applications.